SUMMIT, N.J.--(BUSINESS WIRE)--Celgene Corporation(NASDAQ:CELG) todayannounced it has
fulfilled the accelerated approval requirements for POMALYST®
(pomalidomide) based on results from MM-003, an international phase III
study of POMALYST plus low-dose dexamethasone versus high-dose
dexamethasone in relapsed/refractory multiple myeloma patients.
POMALYST, in combination with dexamethasone is approved for patients
with multiple myeloma who have received at least two prior therapies
including lenalidomide and a proteasome inhibitor and have demonstrated
disease progression on or within 60 days of completion of the last
therapy.

“There remains a significant unmet need for relapsed/refractory multiple
myeloma patients. POMALYST has been able to help thousands of patients
since its approval in 2013 and this data now confirms its survival
benefits,” said Jacqualyn A. Fouse, Ph.D., President, Global Hematology
and Oncology for Celgene. “This label update provides important
information about a key product in our industry-leading portfolio of
therapies for patients with multiple myeloma.”

The pre-specified, final analysis for overall survival (OS) showed a
median OS for the POMALYST plus low-dose dexamethasone arm of 12.4
months (95% CI: 10.4, 15.3), compared to the high-dose dexamethasone arm
of 8 months (95% CI: 6.9, 9.0). This survival benefit was statistically
significant (HR 0.70 [two-sided 95% CI: 0.54, 0.92], p=0.009) even
though 53% of patients in the high-dose dexamethasone arm had
subsequently received POMALYST. The hazard ratio of 0.70 equated to a
30% reduction in the risk of death for patients receiving POMALYST plus
low-dose dexamethasone. Median PFS and OS were based on the assessment
of an Independent Review Adjudication Committee.

POMALYST was initially approved by the FDA in February 2013 under the
agency’s accelerated approval program based on the phase II study,
MM-002.

POMALYST® (pomalidomide) is a thalidomide analogue
indicated, in combination with dexamethasone, for patients with multiple
myeloma who have received at least two prior therapies including
lenalidomide and a proteasome inhibitor and have demonstrated disease
progression on or within 60 days of completion of the last therapy.

POMALYST is contraindicated in pregnancy. POMALYST is a
thalidomide analogue. Thalidomide is a known human teratogen
that causes severe birth defects or embryo-fetal death. In
females of reproductive potential, obtain 2 negative pregnancy
tests before starting POMALYST treatment.

Females of reproductive potential must use 2 forms of
contraception or continuously abstain from heterosexual sex
during and for 4 weeks after stopping POMALYST treatment.

POMALYST is only available through a restricted distribution
program called POMALYST REMS®.

Venous and Arterial Thromboembolism

Deep venous thrombosis (DVT), pulmonary embolism (PE),
myocardial infarction, and stroke occur in patients with
multiple myeloma treated with POMALYST. Prophylactic
antithrombotic measures were employed in clinical trials.
Thromboprophylaxis is recommended, and the choice of regimen
should be based on assessment of the patient’s underlying risk
factors.

CONTRAINDICATIONS: Pregnancy

POMALYST can cause fetal harm and is contraindicated in females who
are pregnant. If POMALYST is used during pregnancy or if the patient
becomes pregnant while taking this drug, the patient should be
apprised of the potential hazard to a fetus

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity

Females of Reproductive Potential: Must
avoid pregnancy while taking POMALYST and for at least 4 weeks after
completing therapy. Must commit either to abstain continuously from
heterosexual sexual intercourse or to use 2 methods of reliable birth
control, beginning 4 weeks prior to initiating treatment with
POMALYST, during therapy, during dose interruptions, and continuing
for 4 weeks following discontinuation of POMALYST therapy. Must obtain
2 negative pregnancy tests prior to initiating therapy

Males: Pomalidomide is present in the
semen of patients receiving the drug. Males must always use a latex or
synthetic condom during any sexual contact with females of
reproductive potential while taking POMALYST and for up to 28 days
after discontinuing POMALYST, even if they have undergone a successful
vasectomy. Males must not donate sperm

Blood Donation: Patients must not donate
blood during treatment with POMALYST and for 1 month following
discontinuation of POMALYST therapy because the blood might be given
to a pregnant female patient whose fetus must not be exposed to
POMALYST

POMALYST REMS® Program

Because of the embryo-fetal risk,POMALYST is available only
through a restricted program under a Risk Evaluation and Mitigation
Strategy (REMS) called “POMALYST REMS®.”
Prescribers and pharmacies must be certified with the program; patients
must sign an agreement form andcomplywith the
requirements. Further information about the POMALYST REMS®
program is available at www.CelgeneRiskManagement.com
or by telephone at 1-888-423-5436.

Hepatotoxicity: Hepatic failure, including fatal cases, has
occurred in patients treated with POMALYST. Elevated levels of alanine
aminotransferase and bilirubin have also been observed in patients
treated with POMALYST. Monitor liver function tests monthly. Stop
POMALYST upon elevation of liver enzymes. After return to baseline
values, treatment at a lower dose may be considered.

Hypersensitivity Reactions: Angioedema and severe dermatologic
reactions have been reported. Discontinue POMALYST for angioedema, skin
exfoliation, bullae, or any other severe dermatologic reactions, and do
not resume therapy.

Dizziness and Confusional State: In trials 1 and 2 in patients
who received POMALYST + Low-dose Dex, 14% experienced dizziness and 7% a
confusional state; 1% of patients experienced Grade 3 or 4 dizziness and
3% experienced a Grade 3 or 4 confusional state. Instruct patients to
avoid situations where dizziness or confusional state may be a problem
and not to take other medications that may cause dizziness or
confusional state without adequate medical advice.

Risk of Second Primary Malignancies: Cases of acute myelogenous
leukemia have been reported in patients receiving POMALYST as an
investigational therapy outside of multiple myeloma.

Tumor Lysis Syndrome: Tumor lysis syndrome (TLS) may occur in
patients treated with POMALYST. Patients at risk are those with high
tumor burden prior to treatment. These patients should be monitored
closely and appropriate precautions taken.

Pomalidomide is primarily metabolized by CYP1A2 and CYP3A. Pomalidomide
is also a substrate for P-glycoprotein (P-gp). Avoid the use of strong
CYP1A2 inhibitors. If medically necessary to co-administer strong
inhibitors of CYP1A2 in the presence of strong inhibitors of CYP3A4 and
P-gp, reduce POMALYST dose by 50%. Cigarette smoking may reduce
pomalidomide exposure due to CYP1A2 induction. Patients should be
advised that smoking may reduce the efficacy of pomalidomide.

USE IN SPECIFIC POPULATIONS

Pregnancy: If pregnancy does occur during treatment, immediately
discontinue the drug and refer patient to an obstetrician/gynecologist
experienced in reproductive toxicity for further evaluation and
counseling. Report any suspected fetal exposure to POMALYST to the FDA
via the MedWatch program at 1-800-332-1088 and also to Celgene
Corporation at 1-888-423-5436.

Nursing Mothers: It is not known if pomalidomide is excreted in
human milk. Pomalidomide was excreted in the milk of lactating rats.
Because many drugs are excreted in human milk and because of the
potential for adverse reactions in nursing infants from POMALYST, a
decision should be made whether to discontinue nursing or to discontinue
the drug, taking into account the importance of the drug to the mother.

Pediatric Use: Safety and effectiveness of POMALYST in patients
under the age of 18 have not been established.

Geriatric Use: No dosage adjustment is required for POMALYST
based on age. Patients >65 years of age were more likely than patients
≤65 years of age to experience pneumonia.

Renal and Hepatic Impairment: Pomalidomide is metabolized in the
liver. Pomalidomide and its metabolites are primarily excreted by the
kidneys. The influence of renal and hepatic impairment on the safety,
efficacy, and pharmacokinetics of pomalidomide has not been evaluated.
Avoid POMALYST in patients with a serum creatinine >3.0 mg/dL. Avoid
POMALYST in patients with serum bilirubin >2.0 mg/dL and AST/ALT >3.0 x
ULN.

MM-003 was a Phase III multi-center, randomized, open-label study where
POMALYST + low-dose dexamethasone therapy was compared to high-dose
dexamethasone in adult patients with relapsed and refractory multiple
myeloma, who had received at least two prior treatment regimens,
including lenalidomide and bortezomib, and demonstrated disease
progression on or within 60 days of the last therapy. For patients
receiving POMALYST + low-dose dexamathasone, 94% were refractory to
lenalidomide, and 74% were refractory to both lenalidomide and
bortezomib. Patients with creatinine clearance ≥ 45ml/min qualified for
the study. A total of 455 patients were enrolled in the study: 302 in
the POMALYST + low-dose dexamethasone arm and 153 in the high-dose
dexamethasone arm. Patients in the POMALYST + low-dose dexamethasone arm
were administered 4 mg POMALYST orally on days 1 to 21 of each 28-day
cycle. Dexamethasone (40 mg) was administered once per day on days 1, 8,
15 and 22 of a 28-day cycle. Patients >75 years of age started treatment
with 20 mg dexamethasone using the same schedule. For the high-dose
dexamethasone arm, dexamethasone (40 mg) was administered once per day
on days 1 through 4, 9 through 12, and 17 through 20 of a 28-day cycle.
Patients >75 years of age started treatment with 20 mg dexamethasone
using the same schedule. Treatment continued until patients had disease
progression.

About POMALYST®

POMALYST is a thalidomide analogue indicated in combination with
dexamethasone for patients with multiple myeloma who have received at
least two prior therapies including lenalidomide and a proteasome
inhibitor and have demonstrated disease progression on or within 60 days
of completion of the last therapy.

Celgene Corporation, headquartered in Summit, New Jersey, is an
integrated global pharmaceutical company engaged primarily in the
discovery, development and commercialization of innovative therapies for
the treatment of cancer and inflammatory diseases through gene and
protein regulation. For more information, please visit www.celgene.com.
Follow Celgene on Twitter @Celgene, and on Pinterest and LinkedIn.

POMALYST® is a registered trademark of Celgene Corporation.

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